Evaluating MY008211A for treating anemia in PNH patients on anti-C5 therapy

A Multicenter, Single-arm, Open-label Phase III Study to Evaluate Efficacy and Safety of MY008211A Tablets in Patients With PNH and Residual Anemia, Despite Treatment With Anti-C5 Antibody.

This study is testing a new treatment called MY008211A to see if it can help people with PNH who still have anemia even while on anti-C5 therapy.

Quick facts

• Phase: Phase 3
• Study type: Interventional
• Enrollment: 20 (estimated)
• Ages: 18 Years to 75 Years
• Sex: All
• Sponsor: Wuhan Createrna Science and Technology Co., Ltd (Industry-sponsored)
• Locations: 1 site (Beijing, Beijing Municipality)
• Trial ID: NCT06932471 on ClinicalTrials.gov

What this trial studies

This phase III clinical trial aims to assess the safety and efficacy of MY008211A in patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) who continue to experience anemia despite being treated with anti-C5 antibodies. The study is multicenter, single-arm, and open-label, meaning all participants will receive the treatment without a control group. Participants must have a confirmed diagnosis of PNH and have been on a stable regimen of anti-C5 therapy for at least six months, with hemoglobin levels remaining below 100 g/L. The trial will monitor the participants' response to MY008211A through various assessments over the treatment period.

Who should consider this trial

Good fit: Ideal candidates are adults aged 18 and older with a confirmed diagnosis of PNH and persistent anemia despite anti-C5 antibody treatment.

Not a fit: Patients with severely low blood cell counts or a history of recurrent infections may not benefit from this treatment.

Why it matters

Potential benefit: If successful, this treatment could significantly improve hemoglobin levels and overall health for PNH patients who have not responded adequately to existing therapies.

How similar studies have performed: While this approach is novel for this specific patient population, similar studies targeting anemia in other conditions have shown promising results.

Eligibility criteria

Inclusion Criteria:

1. Male and female participants ≥ 18 years of age and BMI ≥ 18.0 kg/m2 with a diagnosis of PNH confirmed by high-sensitivity flow cytometry with clone size ≥ 10%.
2. Stable regimen of anti-C5 antibody treatment for at least 6 months before treatment, and Hb was still \< 100 g/L.
3. The average hemoglobin level of at least two tests in 4 months before screening \< 100 g/L.
4. The average hemoglobin level of two tests in the central laboratory during screening \< 100 g/L.
5. Vaccination against Neisseria meningitidis infection is required prior to the start of study treatment. If not received previously, vaccination against Streptococcus pneumoniae and Haemophilus influenzae infections should be given.

Exclusion Criteria:

1. Patients with reticulocytes \<100x10\^9/L; platelets \<30x10\^9/L; neutrophils \<0.5x10\^9/L.
2. History of recurrent invasive infections caused by encapsulated organisms,e.g. meningococcus or pneumococcus.
3. Known or suspected hereditary complement deficiency.
4. Previous bone marrow or hematopoietic stem cell transplantation.
5. Previous splenectomy.
6. A history of malignancy within 5 years before screening, except cured local basal cell carcinoma of the skin and carcinoma in situ of the cervix.

Where this trial is running

Beijing, Beijing Municipality

• Peking Union Medical College Hospital (PUMCH). — Beijing, Beijing Municipality, China (Recruiting)

Study contacts

• Study coordinator: Wuhan Createrna Science and Technology Co.,Ltd

Conditions Paroxysmal Nocturnal Hemoglobinuria